PMID- 30260711 OWN - NLM STAT- MEDLINE DCOM- 20190409 LR - 20211204 IS - 1941-837X (Electronic) IS - 1369-6998 (Linking) VI - 21 IP - 12 DP - 2018 Dec TI - Cost-effectiveness of axicabtagene ciloleucel for adult patients with relapsed or refractory large B-cell lymphoma in the United States. PG - 1238-1245 LID - 10.1080/13696998.2018.1529674 [doi] AB - PURPOSE: Axicabtagene ciloleucel (axi-cel) was recently approved for treatment of relapsed or refractory (R/R) large B-cell lymphoma (LBCL) following two or more prior therapies. As the first CAR T-cell therapy available for adults in the US, there are important questions about clinical and economic value. The objective of this study was to assess the cost-effectiveness of axi-cel compared to salvage chemotherapy using a decision model and a US payer perspective. MATERIALS AND METHODS: A decision model was developed to estimate life years (LYs), quality-adjusted life years (QALYs), and lifetime cost for adult patients with R/R LBCL treated with axi-cel vs salvage chemotherapy (R-DHAP). Patient-level analyses of the ZUMA-1 and SCHOLAR-1 studies were used to inform the model and to estimate the proportion achieving long-term survival. Drug and procedure costs were derived from US average sales prices and Medicare reimbursement schedules. Future healthcare costs in long-term remission was derived from per capita Medicare spending. Utility values were derived from patient-level data from ZUMA-1 and external literature. One-way and probabilistic sensitivity analyses evaluated uncertainty. Outcomes were calculated over a lifetime horizon and were discounted at 3% per year. RESULTS: In the base case, LYs, QALYs, and lifetime costs were 9.5, 7.7, and $552,921 for axi-cel vs 2.6, 1.1, and $172,737 for salvage chemotherapy, respectively. The axi-cel cost per QALY gained was $58,146. Cost-effectiveness was most sensitive to the fraction achieving long-term remission, discount rate, and axi-cel price. The likelihood that axi-cel is cost-effective was 95% at a willingness to pay of $100,000 per QALY. CONCLUSION: Axi-cel is a potentially cost-effective alternative to salvage chemotherapy for adults with R/R LBCL. Long-term follow-up is necessary to reduce uncertainties about health outcomes. FAU - Roth, Joshua A AU - Roth JA AD - a Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Institute , Seattle , WA , USA. AD - b Comparative Health Outcomes, Policy and Economics Institute, School of Pharmacy , University of Washington , Seattle , WA , USA. FAU - Sullivan, Sean D AU - Sullivan SD AD - a Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Institute , Seattle , WA , USA. AD - b Comparative Health Outcomes, Policy and Economics Institute, School of Pharmacy , University of Washington , Seattle , WA , USA. FAU - Lin, Vincent W AU - Lin VW AD - c Kite, a Gilead Company , Santa Monica , CA , USA. FAU - Bansal, Aasthaa AU - Bansal A AD - a Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Institute , Seattle , WA , USA. AD - b Comparative Health Outcomes, Policy and Economics Institute, School of Pharmacy , University of Washington , Seattle , WA , USA. FAU - Purdum, Anna G AU - Purdum AG AD - c Kite, a Gilead Company , Santa Monica , CA , USA. FAU - Navale, Lynn AU - Navale L AD - c Kite, a Gilead Company , Santa Monica , CA , USA. FAU - Cheng, Paul AU - Cheng P AD - c Kite, a Gilead Company , Santa Monica , CA , USA. FAU - Ramsey, Scott D AU - Ramsey SD AD - a Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Institute , Seattle , WA , USA. AD - b Comparative Health Outcomes, Policy and Economics Institute, School of Pharmacy , University of Washington , Seattle , WA , USA. LA - eng PT - Comparative Study PT - Journal Article DEP - 20181016 PL - England TA - J Med Econ JT - Journal of medical economics JID - 9892255 RN - 0 (Antigens, CD19) RN - 0 (Biological Products) RN - U2I8T43Y7R (axicabtagene ciloleucel) SB - IM EIN - J Med Econ. 2018 Dec;21(12):1255. PMID: 30485139 CIN - J Med Econ. 2019 Jul;22(7):613-615. PMID: 30747012 MH - Antigens, CD19/adverse effects/economics/*therapeutic use MH - Antineoplastic Combined Chemotherapy Protocols/adverse effects/economics/*therapeutic use MH - Biological Products MH - Cost-Benefit Analysis MH - Decision Support Techniques MH - Fees, Pharmaceutical/statistics & numerical data MH - Health Expenditures/statistics & numerical data MH - Humans MH - Immunotherapy, Adoptive/adverse effects/economics/*methods MH - Lymphoma, B-Cell/*drug therapy/mortality MH - Models, Econometric MH - Quality-Adjusted Life Years MH - Recurrence MH - Salvage Therapy/economics MH - Survival Analysis MH - United States OTO - NOTNLM OT - Axicabtagene ciloleucel OT - C44 OT - C53 OT - CAR T-cell therapy OT - cost-effectiveness OT - lymphoma EDAT- 2018/09/28 06:00 MHDA- 2019/04/10 06:00 CRDT- 2018/09/28 06:00 PHST- 2018/09/28 06:00 [pubmed] PHST- 2019/04/10 06:00 [medline] PHST- 2018/09/28 06:00 [entrez] AID - 10.1080/13696998.2018.1529674 [doi] PST - ppublish SO - J Med Econ. 2018 Dec;21(12):1238-1245. doi: 10.1080/13696998.2018.1529674. Epub 2018 Oct 16.